• SEC Filings

ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE) Files An 8-K Other Events
Item 8.01

On August 1, 2019, Ultragenyx Pharmaceutical Inc. issued a press release announcing that it has submitted to the U.S. Food and Drug Administration a New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (the “Press Release”). A copy of the Press Release is filed as Exhibit 99.1

(d) Exhibits

Ultragenyx Pharmaceutical Inc. Exhibit
EX-99.1 2 rare-ex991_37.htm EX-99.1 rare-ex991_37.htm Exhibit 99.1   Contact: Ultragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 [email protected]   Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders   Novato,…
To view the full exhibit click here

About ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE)

Ultragenyx Pharmaceutical Inc. is a clinical-stage biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The Company is engaged in the identification, acquisition, development and commercialization of products for the treatment of rare and ultra-rare diseases segment. Its pipeline consists of two product categories: biologics, including a monoclonal antibody and enzyme replacement therapies, and small-molecule substrate replacement therapies. KRN23 is a fully human monoclonal antibody administered via subcutaneous injection. Recombinant human beta-glucuronidase (rhGUS), is an intravenous (IV), enzyme replacement therapy for the treatment of mucopolysaccharidosis 7 (MPS 7). It is developing UX007 for oral administration intended as a substrate replacement therapy. It is developing aceneuramic acid extended-release (Ace-ER) for the treatment of GNE myopathy.